Sensorineural hearing loss, the most common form of hearing impairment, is mainly attributable to genetic mutations or acquired factors, such as aging, noise exposure, and ototoxic drugs. In the field of gene therapy, advances in genetic and physiological studies and profound increases in knowledge regarding the underlying mechanisms have yielded great progress in terms of restoring the auditory function in animal models of deafness. Nonetheless, many challenges associated with the translation from basic research to clinical therapies remain to be overcome before a total restoration of auditory function can be expected. In recent years, Chinese research teams have promoted various developmental efforts in this field, including gene sequencing to identify additional potential loci that cause deafness, studies to elucidate the underlying molecular mechanisms, and research to optimize vectors and delivery routes. In this review, we summarize the state of the field and focus mainly on the progress of gene therapy in animal model studies and the optimization of therapeutic strategies in China.
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This work was supported by National Natural Science Foundation of China (No. 81530029, 81720108010, 81800919, and 81971240) and by Shanghai Health System (No. 2017YQ010).
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Zhang, Z., Wang, J., Li, C. et al. Gene therapy development in hearing research in China. Gene Ther 27, 349–359 (2020). https://doi.org/10.1038/s41434-020-0177-1
Gene Therapy (2020)